Stem Cell Clinic

Unproven Stem Cell Treatments Offer Hope & Risks

The injections of stem cells into his spine were supposed to help Jim Gass, 66, recover from a stroke he had six years ago.

Gass traveled to clinics in Mexico, China, and Argentina to undergo these unproven procedures. Including travel, he spent close to $300,000, according to a story in The New York Times.

After the final round of shots, he was able to walk better. But his hope for a full recovery was cut short. While on vacation in Thailand six months after his treatments, he developed low back pain and difficulty walking and standing.

Back in Boston, doctors at Brigham and Womens Hospital did an MRI scan of his spine, and found a large mass filling the entire lower part of his spinal column.

Genetic testing revealed that the abnormal, primitive cells of the mass did not come from Gass, but from stem cells injected into his spine.

Radiation treatments seemed to slow the growth of the mass and improve Gass symptoms. But another scan done later in San Diego showed that the mass was growing again.

The doctors involved wrote about his case in a letter published June 22 in the New England Journal of Medicine.

Despite the outcome of this case, experts familiar with this kind of stem cell tourism say that some good may still come of it.

It is a really sad case, but its good that its causing discussion around both the potential harm of these therapies and the lack of evidence regarding the benefits, Timothy Caulfield, research director of the Health Law Institute at the University of Alberta, who wrote a recent commentary on stem cell hype, told Healthline.

Read more: Get the facts on stem cell research

This is not the first time that stem cell treatments have led to bad outcomes such as tumors or lesions.

There have been other reports of adverse events as a result of these kinds of therapies, said Caulfield. There have even been reports of adverse events when the procedure is less extreme such as people getting stem cell therapy for anti-aging, anti-wrinkle procedures.

Caulfield is quick to point out that therapy should be in quotes because with the exception of a few approved treatments the use of stem cells to treat illnesses has not reached the point where it is ready for widespread use in clinics.

There are very few stem cell therapies that have been proven, at this point, to be efficacious, said Caulfield. Lots of exciting work is going on theyre in clinical trials right now but for most conditions we simply arent there yet.

Although there are a few documented cases like Gass, many more may go unreported, resulting from treatments at unregulated stem cell clinics around the world.

We dont know exactly how many people are having these procedures, Dr. Jaime Imitola, a neurologist and stem cell researcher at The Ohio State University Wexner Medical Center, who has written about the dangers of stem cell tourism and how to counsel patients, told Healthline.

There are so many diseases that these clinics are often treating for from diabetes to ALS and some of these treatments may involve more risk than others, Imitola said.

Theres a big difference in risk between taking cells from your own body and putting them back in your blood, and injecting foreign cells into your spine, as was done in Gass case.

Also, these clinics are not part of a clinical research program, so there are a lot of unknowns about what happens during the procedures.

Are they actually using stem cells? How are they getting the stem cells into people? said Caulfield. Those are all open questions, because it is such an unregulated field.

While Gass traveled outside the United States for injections, unproven stem cell therapies show up much closer to home.

A paper published online Thursday in the journal Cell Stem Cell found that at least 351 businesses in the United States are marketing stem cell therapies that have not gone through the rigorous clinical trial process, or been approved by the Food and Drug Administration (FDA).

These businesses marketed stem cells as treatment for a wide range of conditions from spinal cord injuries and immune system problems to heart disease or even cosmetic fixes.

Read more: Stem cell treatments offering hope for MS patients

With few treatments available for many diseases, stem cell clinics step in to fill the void, many overhyping the actual research being done in this area.

[Clinics] are leveraging excitement around legitimate stem cell research and the pop culture footprint Ill put it that way of stem cells, said Caulfield.

Some of this hype has been generated when high-profile athletes undergo stem cell therapy and see improvements, like Peyton Manning did in Germany for a neck injury.

The company that Gass contacted had been involved in the treatment of former NFL quarterback John Brodie.

These remarkable success stories offer people hope. But because they happened outside a clinical trial, its impossible to know if the athletes health would have improved on their own.

Imitola compares this to using acupuncture alongside proven treatments.

If I give you acupuncture after a stem cell treatment, I cannot make the distinction whether what happens is a result of the acupuncture or the treatment, said Imitola, because this is not a clinical trial.

Researchers, universities, and the media also have a hand in stem cell hype. The time element, in particular, can be misrepresented.

"I think that the scientific community really needs to be careful how they talk about stem cell research, said Caulfield. We did a study that showed, for example, that the time from doing basic research to getting into the clinic is often exaggerated when people talk about stem cell research. Our study found that it was often portrayed as if the research was going to be in the clinic in 5 to 10 years, or sooner, which is really, really fast. It creates unrealistic expectations."

Read more: Stem cells as a possible treatment for rheumatoid arthritis

Patients with spinal cord injuries or diseases are often anxious for new treatments to be approved quickly. But stem cell researchers have good reason to be cautious.

One characteristic that stem cells share with cancer cells is that they both multiply rapidly. This is why stem cell researchers have long been concerned that stem cells could form tumors.

Thats why there are so many years of testing in the lab, in animal models, and finally in clinical trials.

It is unethical to offer a procedure or a drug that is unproven, said Imitola.

When clinics skip ahead and offer treatments that have not been properly tested, they may end up hurting people instead of helping them.

Its interesting because [Gass] case, and others, is generating a new disease, a new complication, an iatrogenic tumor, said Imitola.

Of course, bad outcomes can happen during a clinical trial. But these are tracked, and clinical trials can be shut down if unforeseen side effects happen.

A recent stem cell clinical trial in Japan was stopped, because when the researchers looked at whether the cells were clean from a genetic point of view, the cells had some problems, some changes, said Imitola, So the researchers said, We cant do that, we cant inject the cells.

Imitola recently co-authored a paper in JAMA Neurology calling on doctors to educate patients with neurological diseases about stem cell tourism.

But he admits that cases like Gass can serve as an even more effective warning.

This patient, in particular, is important because he put a human face to this tragedy, said Imitola. We need more patients to come forward. Most likely, this is not an isolated case.

Read more: Stem cell treatment for COPD

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Unproven Stem Cell Treatments Offer Hope & Risks

Update on stem cell treatment cost for 2018 from ongoing poll …

I get asked many questions about stem cell therapies, but one of the most common over the years has been about the stem cell treatment cost. For instance, a reporter might ask, How much does a stem cell treatment for MS cost? and a patient might ask me, How much is a fair cost for a stem cell therapy for arthritis? Or, patients will voluntarily tell me what they paid or mention it in the comments. We hear various numbers thrown around about costs so I decided to do a poll on this. I even did an early update on the results of this poll, voicing my skepticism that the costs paid were worth it.

But the poll has gotten well over 500 responses now so I thought I would revisit it and what it might mean.

You can see a screenshot of the images. Its fair to say, as much as Internet polls arent considered particularly accurate, that this one largely fits with what is reported out in the field.

(On a side note, I wish there was such a thing as going out into the field for stem cell scientists as Ive always been a bit jealous of scientists who really do go out in the field. What do we do, go out in the wild and catch wild or feral stem cells in the bush?)

Patients self-reported most often paying between $2,500 and $7,500 for their stem cell therapy so if we take the average of those we get that $5,000 figure that is what I hear most often from others. Yes, not necessarily very rigorous, but the result makes good sense. Not far behind though were responses in the $7,500-20,000 range.

About 1 in 10 respondents reported paying $20,000 or more, including some beyond $100,000. Thats a whopping stem cell treatment cost, especially for something most often unproven and unapproved by the FDA.

If we consider these responses, the average cost may be more like $7,500-$10,000.

Notably, about 1/16 respondents indicated their stem cells were free. Im not sure what that means in terms of how that came to be.

Interestingly, most respondents who also went on to answer a 2nd poll in that post about where they got the treatment indicate it was at a stem cell clinic (scroll down in that Oct. 2017 post and youll see the 2nd poll). This 2nd poll has about 200 responses.

So today buying a simple stem cell treatment, most often unproven and non-FDA approved, is often not so different in cost than buying a 10-year old used car, while less often it is similar to buy various new cars including at the high end of stem cell therapy cost, some very expensive new cars. This cost and the risks involved are why I have suggested to patients in the past to be assertive when considering a stem cell treatment, ask questions, dont just accept too good to be true kinds of answers, etc. In short, be at least (or ideally much more) rigorous about unproven stem cell treatments as you are about buying a car.

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Hematology society recognizes four School of Medicine researchers Washington University School of Medicine in St. Louis – Washington University…

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ASH Scholar Awards support fellows to junior faculty in hematology

The American Society of Hematology has recognized four Washington University researchers with ASH Scholar Awards, supporting early-career faculty whose research is focused on understanding and treating blood disorders. Clockwise from top left: Amanda Smith, PhD, Julia Warren, MD, PhD, Stephanie Luff, PhD, and Karolyn Oetjen, MD, PhD.

Four early-career researchers at Washington University School of Medicine in St. Louis have been recognized by the American Society of Hematology (ASH) for their dedication to the field of hematology. The ASH Scholar Awards provide financial support to fellows and junior faculty who have dedicated their careers to the study of blood disorders.

One of ASHs most prestigious research award programs, the Scholar Awards provide funding to young scientists as they make the transition from training programs to careers leading independent labs.

Karolyn A. Oetjen, MD, PhD, Amanda M. Smith, PhD, Stephanie Luff, PhD, and Julia T. Warren, MD, PhD, are among the 39 recipients of the 2020 ASH Scholar Awards. Depending on whether the researcher is a fellow or junior faculty member, the awards provide $100,000 to $150,000 over a two- to three-year period to support basic, translational and clinical research seeking to understand and improve the treatment of blood disorders.

Oetjen, an instructor in medicine, uses single-cell sequencing and imaging techniques to understand how cancer cells evolve and adapt to chemotherapy, allowing the recurrence of blood cancers including acute myeloid leukemia (AML) and myelodysplastic syndromes.

Smith, an instructor in medicine, studies the origins of AML in the lab of Timothy J. Ley, MD, the Lewis T. and Rosalind B. Apple Professor of Oncology. She is interested in understanding the events that lead a normal cell to start showing the early signs of becoming cancerous and, from there, identifying the triggers that lead to full-blown AML. The goal is to identify strategies to block this process.

Luff is a postdoctoral research scholar in the lab of Christopher M. Sturgeon, PhD, an assistant professor of medicine. She studies blood-forming stem cells in the early embryo in an effort to understand how they develop.

Warren is a fellow in pediatric hematology/oncology, conducting postdoctoral research in the lab of Daniel C. Link, MD, the Alan A. and Edith L. Wolff Professor of Medicine. Her work is focused on understanding the genes and metabolic processes that regulate formation of granulocytes, a type of white blood cell. Understanding these processes could help develop new treatments for patients with diseases that affect granulocytes and for cancer patients whose treatment causes low numbers of white blood cells, increasing the risk of infection.

The American Society of Hematology is the worlds largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment and prevention of disorders affecting the blood.

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Hematology society recognizes four School of Medicine researchers Washington University School of Medicine in St. Louis - Washington University...

After pursuing him two years ago, the Padres are betting on Tommy Pham – The Athletic

PEORIA, Ariz. Two summers ago, members of the Padres front office trekked to Cooperstown, N.Y., to attend the enshrinement of one of their own. They watched as Trevor Hoffman, franchise icon and senior advisor, was inducted into the National Baseball Hall of Fame. They toasted and they celebrated. And, in between festivities, they tapped away at their phones.

In a wooded, secluded region of Central New York, team executives passed the final hours before the July 2018 trade deadline. Less than two weeks earlier, the Padres had sent relievers Brad Hand and Adam Cimber to Cleveland for catching prospect Francisco Meja. Now they were attempting, among other things, to acquire a St. Louis Cardinals outfielder named Tommy Pham.

We were going back and forth, general manager A.J. Preller said.

At the time, the Padres were slogging through the third year of a rebuild. They had signed first baseman Eric Hosmer to a nine-figure contract, but not six...

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After pursuing him two years ago, the Padres are betting on Tommy Pham - The Athletic

The Art of Science Cutting edge science and captivating images – The – The Institute of Cancer Research

Image: The winning image by PhD student Sumana Shrestha, showing differentiating brain cancer cells used to study glioblastoma

PhD student Sumana Shrestha at The Institute of Cancer Research, London, has won the ICRs Science and Medical imaging competition with a colourful image of neural stem cells to study glioblastoma, as well as the public vote, with 212 votes for her detailed image of microparticles.

Researchers from the ICR and The Royal Marsdensubmitted some fantastic images to this years competition, with a wide array of images that tell vivid stories about our science.

Entries to the competition have all been created in the course of our pioneering cancer research but at times they can look like a piece of modern art, and they each tell a compelling story about our research.

The most engaging and exciting image selected by our panel of judges is named the winner of the ICR Science and Medical Imaging Competition.

Our followers on social media picked the supporters' favourite through a public vote, with over 800 votes submitted across Facebook, Instagram and Twitter.

The winning image was taken using a technique called confocal microscopy and shows neural stem cells from mice which are being used to study glioblastoma, an aggressive type of brain cancer. The cells were genetically modified and exposed to a virus to develop glioblastoma, before mice were treated with microRNA repressors and activators to change how the cells grow in the brain and specialise.

A process called immunostaining shows up a garden of stem cells growing and specialising into specific types of cell, to carry out jobs in the brain. Star shaped cells called astrocytes are marked in green, which perform a range of functions in the brain, while neurons are marked in red, cells in the nervous system that help transmit information.

MicroRNAs are proteins in cells that help regulate gene expression, and in cancer they can be severely malfunctioning. Modulating microRNA levels in brain cancer cells could have a major impact on how the tumour cells develop, which could help to make the disease less aggressive and easier to treat.

The judges picked this as their wining image noting the striking use of colours in the image, and how Sumana has used her image to illustrate how her research is helping to tackle cancer.

Catherine Graham, Head of Brand and Creative at the ICR, said:

"Sumanas image is really eye-catching and it is a great example of the technical skill and creative flair of our researchers, and the visual impact that can be captured even in a disease like cancer.

Choosing the winning image from all of those submitted to the competition this year was challenging, but it was a pleasure to see the variety of our researchers work and to learn more about their science.

PhD student Sumana Shrestha said:

This is very unexpected news for me as the competition was so strong, but I am over the moon to find that my image has won! Many thanks to the judges for taking their time to go through the images. I am very excited for the announcement, and again, overjoyed about the results.

The image shows microparticles, tiny beads about the width of a human hair, taken using scanning electron microscopy. To image the microparticles, they are first coated in a layer of gold just nanometres thick to make them conductive. The particles have been etched using an antibiotic called fusidic acid to create dimples on their surface like a golf-ball.

Microparticles can be used to deliver drugs in the body, but studies have shown that the textured surface of microparticles can also have an effect on cell behaviour.

Cells are influenced by the topographical features of their surroundings, and the 'dimples' on the treated microparticles provide an environment that can be used to control proliferation or differentiation of stem cells. Manipulating fundamental cellular processes using microparticles could have huge implications in cancer, as well as applications in regenerative medicine.

This image received a total of 212 across the ICRs social media channels to be crowned our supporters favourite, with an ICR follower on social media thinking the microparticles looked like pollen grains.

Science Communications Officer Graham Shaw, who organised the competition, said: Its surprising that two submissions from one researcher have won both prizes in the competition, but this black and white image clearly captured the imaginations of our supporters on social media.

The detail on the microparticles is amazing, and their potential to alter cells as a way to treat cancer is a really powerful idea. This image helps to convey how this has been implemented in cancer research.

Eight of the most highly rated and vibrant images from this year's competition were voted on by our supporters for their favourite image. Learn more about the other amazing images that made it onto our shortlist.

The eye of the Storm: the tumultuous yet spectacular nature of cancer tumours by Dr Lisa Pickard and Louise Howell

The eye of the storm: the tumultuous, dramatic nature of cancer tumours. Lab grown mini-tumours are better at capturing the unique characteristics of tumours in patients, and can be used to understand how they might respond to cancer drugs.

Melanoma on a Chip by Professor Chris Bakal and Nick Moser at Imperial College, London

Melanoma on a chip: part of a melanoma cell has been blasted away using a technique called ion beam milling. This process helps researchers look inside cells in unprecedented detail, to help them understand the processes happening inside.

Melanoma cell invasion in 3D by Vicky Bousgouni, Professor Chris Bakal and David Robertson

Invading melanoma cell in 3D. A cell from aggressive skin cancer is growing into and around fibres of collagen, mimicking how cancer invades tissue. Cancer remodels its environment to spread around the body, so understanding this process could make it easier to treat.

Super-resolution microscopy image of cell focal adhesions by PhD Student Ian Jones, Dr Lucas Dent, and Professor Chris Bakal

The first ever super-resolution microscopy image taken of focal adhesions molecules inside cellular structures which help cancer cells move and spread around the body.

The Art of Deception by Dr Jarama Clucas

The Art of Deception. These aggressive breast cancer cells look like structures normally seen in healthy breast tissue. Cancer use deception to hide from the immune system and avoid treatment, but growing samples in the lab can be used to test treatments that could help our body fight the disease.

Forward and reverse translation by PhD Student Somaieh Hedayat and Louise Howell

Tumour organoids are mini replicas of a patients tumours, and could be used to rapidly test a patients cancer against a range of cancer drugs to see which ones may be most effective.

These eye-catching images illustrate just some of the cutting-edge research being carried out at the ICR, taken using sophisticated equipment purchased thanks to generous donations from our supporters.

From images like these our researchers are gaining unprecedented insights into the mechanisms that drive cancer, and new ways to target the disease to help treat patients.

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The Art of Science Cutting edge science and captivating images - The - The Institute of Cancer Research

Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine – Endpoints News

In August of 2011, Carl June and his team published a landmark paper showing their CART treatment had cleared a patient of cancer. A year-to-the-month later, Jennifer Doudna made an even bigger splash when she published the first major CRISPR paper, setting off a decade of intense research and sometimes even more intense public debate over the ethics of what the gene-editing tool could do.

Last week, June, whose CART work was eventually developed by Novartis into Kymriah, published in Sciencethe first US paper showing how the two could be brought together. It was not only one of the first time scientists have combined the groundbreaking tools, but the first peer-reviewed American paper showing how CRISPR could be used in patients.

June used CRISPR to edit the cells of three patients with advanced blood cancer, deleting the traditional T cell receptor and then erasing the PD1 gene, a move designed to unleash the immune cells. The therapy didnt cure the patients, but the cells remained in the body for a median of 9 months, a major hurdle for the therapy.

Endpoints caught up with June about the long road both he and the field took to get here, if the treatment will ever scale up, and where CRISPR and other advancements can lead it.

The interview has been condensed and edited.

Youve spoken in the past about howyou started working in this field in the mid-90s after your wife passed away from cancer. What were some of those early efforts? How did you start?

Well, I graduated from high school and had a low draft number [for the Vietnam War] and was going to go to study engineering at Stanford, but I was drafted and went into the Naval Academy in 1971, and I did that so I wouldnt have to go to the rice fields.

The war ended in 73, 74, so when I graduated in 1975, I was allowed to go to medical school, and then I had a long term commitment to the Navy because they paid for the Acadamy and Medical school. And I was interested in research and at the time, what the Navy cared about was a small scale nuclear disaster like in a submarine, and like what happened at Chernobyl and Fukushima. So they sent me to the Fred Hutchinson Cancer Center where I got trained in cancer, as a medical oncologist. I was going to open a bone marrow transplant center in Bethesda because the Navy wanted one in the event of a nuclear catastrophe.

And then in 1989, the Berlin Wall came down and there was no more Cold War. I had gone back to the Navy in 86 for the transplant center, which never happened, so then I had to work in the lab full time. But in the Navy, all the research has to be about combat and casualty. They care about HIV, so my first papers were on malaria and infectious disease. And the first CAR-T trials were on HIV in the mid-90s.

In 96, my wife got diagnosed with ovarian cancer and she was in remission for 3-4 years. I moved to the University of Pennsylvania in 1999 and started working on cancer because I wasnt allowed to do that with the Navy. My wife was obviously a lot of motivation to do that. She passed away in 2001. Then I started working with David Porter on adoptive transfer T cells.

I got my first grant to do CAR-T cells on HIV in 2004, and I learned a whole lot. I was lucky to have worked on HIV because we did the first trials using lentiviruses, which is an engineered HIV virus.

I was trained in oncology, and then because of the Navy forced to work on HIV. It was actually a blessing in disguise.

So if you hadnt been drafted, you wouldve become an engineer?

Yes. Thats what I was fully intending. My dad was a chemical engineer, my brother is an engineer. Thats what I thought I was going to do. No one in my family was ever a physician. Its one of those many quirks of fate.

Back then, we didnt have these aptitude tests. It was just haphazard. I applied to three schools Berkeley, Stanford and Caltech and I got into all three. It was just luck, fate.

And it turned out when I went to the Naval Academy, they had added a pre-med thing onto the curriculum the year before, so thats what I did when I started, I did chemistry.

I wouldve [otherwise] been in nuclear submarines. The most interesting thing in the Navy then was the nuclear sub technology.

You talked about doing the first CAR-T trials on HIV patients because thats where the funding was. Was it always in your head that this was eventually going to be something for cancer?

So I got out of the Navy in 99 and moved to Penn. I started in 98 working on treating leukemia, and then once I got to Penn, I continued working one day a week on HIV.

Its kind of a Back-to-the-Future thing because now cancer has paved out a path to show that CART cells can work and put down the manufacturing and its going to be a lot cheaper making it for HIV. I still think thats going to happen.

Jim Riley, who used to be a postdoc in my lab, has some spectacular results in monkeys with HIV models. They have a large NIH and NIAID research program.

So were going to see more and more of that. The CAR technology is going to move outside of cancer, and into autoimmune and chronic infections.

I want to jump over to cytotoxic release syndrome (CRS)because a big part of the CRISPR study was that it didnt provoke this potentially deadly adverse effect. When did you first become aware that CRS was going to be a problem?

I mean we saw it in the very first patient we treated but in all honesty, we missed it. Im an MD, but I dont see the patient and David Porter tookcare of the first three patients and our first pediatric patient,Emily Whitehead.

In our first patients, 2 out of 3, had complete remission and there were fevers and it was CRS but we thought it was just an infection, and we treated with antibiotics for 3 weeks and[eventually] it went away. And sort of miraculously he was in remission and is still in remission, 9 years later.

And then when we treated Emily. She was at a 106-degree fever over three days, and there was no infection.

Ive told this story before. My daughter has rheumatoid arthritis, and I had been president of the Clinical Immunologists Society from 2009 to 2010, and the first good drug for juvenile rheumatoid arthritisthat came out. I was invited to give the Japanese scientist Tadamitsu Kishimoto the presidential award for inventing the drug.

Then in 2012, Emily Whitehead was literally dying from CRS, she had multiple organ failures. And her labs came back and IL-6 levels were 1000x normal. It turns out the drug I was looking at for my daughter, it blocks IL-6 levels. I called the physician and I said, listen theres something actionable here, since its in your formulary to give it to her off-label.

And she gave her the appropriate dose for rheumatoid arthritis. It was miraculous. She woke up very rapidly.

Now its co-labeled. When the FDA approvedKymriah, it was co-labeled. It kind of saved the field.

How were you feeling during this time? Did you have any idea what was happening to her?

No, not until we got the cytokine levels, and then it was really clear. The cytokine levels go up and it exactly coincided. Then we retroactively checked out adults and they had adverse reactions and it easy to see. We hadnt been on the lookout because it wasnt in our mouse models.

And it appeared with those who got cured. Its one of the first on-target toxicities seen in cancer, a toxicity that happens when you get better. All the toxicities from chemotherapy are off-target: like leukopenia or hair loss.

I had a physician who had a fever of 106, I saw him on a fever when he was starting to get CRS. When the nurse came in and it said 106, they thought the thermometer must be broken. On Monday, I saw him, and said how are you feeling and he said fine. And I looked at the thermometer and histemperature was still 102.

People will willingly tolerate on-target toxicity thats very different from chemotherapy if they know it helps get them better. Thats a new principle in cancer therapy.

You had these early CART results almost at the same time that Doudna publishes the first CRISPR papers, then still in bacteria. When did you first start thinking about combining the two?

Yeah, it was published inSciencein 2012 and thats when Emily Whitehead got treated. Its an amazing thing.

Thats something so orthogonal. You think how in the heck can that ever benefit CART cells? but my lab had done the first edited cells in patients, published in 2012. And we used zinc-fingered nucleases, which were the predecessors to CRISPR. It knocked out one gene at a time, but we showed it was safe.

I was already into gene editing because it could make T cells resistant to HIV. So it was pretty obvious that there were candidates in T cells that you can knock out. And almost every lab started working on some with CRISPR, cause it was much easier.

We were the first to get full approval by the FDA, so we worked on it from 2012, had all the preclinical data by 2016, and then it takes a while to develop a lot of new assays for this as we were very cautious to optimize safety and it took longer than we wanted, but in the end, we learned a tremendous amount.

So what did we learn?

First of all our patients had advanced metastatic cancer and had had a lot of chemotherapy. The first patient had had 3 bone marrow transplants.

One thing is feasibility: could you really do all the complex engineering? So we found out we could. feasibility was passed.

Another was the fact that cas9 came out of bacteria, forms of strep and staph. Everyone has pre-existing immunity to Cas9 and we had experience from the first trial with Sangamo[with zinc-finger nucleases] where some patients had a very high fever. In that case, we had used adenoviruses, and it turned out our patients had very high levels of baseline immune response to adenoviruses, so we were worried that would happen with CRISPR, and it did not happen.

It did not have any toxicity. If it had, it would have really set the field back. If there was animmune response to cas9 and CRISPR, there couldve been a real barrier to the field.

And then, the cells survived in the patients. The furthest on, it was 9 months. The cells had a very high level of survival. In the previous trials, the cells survived less than 7 days. In our case, the half-life was 85 days. We dont know the mechanism yet.

And we found very big precision in the molecular scissors, and that was a good thing for the field. You could cut 3 different genes on 3 different chromosomes and have such high fidelity.

It [CRISPR] is living up to the hype. Its going to fix all these diseases.

Whats the potential in CAR-T, specifically?

Well theres many many genes that you can add. There are many genes that knocking outwill make the cells work better. We started with the cell receptor. There are many, I think, academics and biotechs doing this now and it should make the cells more potent and less toxic.

And more broadly, what else are you looking at for the future of CART? The week before your paper, there were the results from MD Anderson on natural killer cells.

Different cell types, natural killer cells, stem cells putting CAR molecules into stem cells, macrophages. One of my graduate students started a company to do CAR macrophages and macrophages actually eat tumor cells, as opposed to T cells that punch holes in them.

There will be different cell types and there will be many more ways to edit cells. The prime editing and base editing. All different new variations.

Youve talked about how people used to think the immuno-oncology, if it ever worked, would nevertheless be a boutique treatment. Despite all the advancements, Novartis and Gilead still have not met the sales they once hoped to grab from their CART treatments. Are you confident CART will ever be widely accessible?

Oh yeah, Novartis sales are going up. They had a hiccup launching.

Back in 96 or 97, when Genentech launched Herceptin, their commercial antibody, they couldnt meet the demand either and then they scaled up and learned how to do better cultures. So right now Novartis is using tech invented in my lab in the 1990s culture tech thats complex and requires a lot of labor, so the most expensive part is human labor. A lot can be made robotic. The scale problem will be much easier.

Thats an engineering problem that will become a thing of the past. The manufacturing problem will get a lot cheaper. Here in the US, we have a huge problem with how drugs are priced. We have a problem with pricing. Thats a political issue.

But in cell therapy, its just kind of the growth things you see in a new industry. Itll get worked out.

This article has been updated to reflect that Jim Riley conducted work on CAR in HIV.

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Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine - Endpoints News

CytoDyn Reports Continued Positive Clinical Data on its Phase 1b/2 mTNBC and Expanded Access Studies for MBC Ahead of Breakthrough Therapy Designation…

VANCOUVER, Washington, Feb. 14, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today continued positive data for its mTNBC and MBC patients.

Metastatic triple-negative breast cancer (mTNBC), an aggressive histological subtype, has a poor prognosis. In addition, metastatic breast cancer (MBC) is breast cancer that has spread beyond the breast and lymph nodes to other organs in the body (typically the bones, liver, lungs, or brain). Both types of cancer pose significant challenges for patients due to their aggressiveness and limited treatment options. An integral part of CytoDyns mission and purpose is to provide effective therapeutic solutions to these patients. Results of the first five patients are as follows:

Patient #1: Enrolled in mTNBC Phase 1b/2 - Injected on 9/27/2019. CTC (circulating tumor cells) dropped to zero in two weeks on 10/11/2019. Total CTC and EMT (Epithelial Mesenchymal Transition in Tumor Metastasis) dropped to zero after about one month of treatment with leronlimab (once-a-week 350 mg dose). After approximately four months of treatment with leronlimab and Carboplatin, the patient had zero CTC+EMT. Furthermore, the patients CT scan indicated a 20% tumor shrinkage within the first few weeks of treatment with leronlimab.

Patient #2: Enrolled in single IND. Patient is MBC with HER2+ stage 4 metastasis to lung, liver, and brain. Patients radiologist cancelled 2nd round of treatment due to leronlimabs effect on shrinking the largest tumor in the brain by 56% and other lesions being stable. Leronlimab has, and continues to be, the only treatment in place since the measurement of brain tumor shrinkage was initiated. Patient was permitted to obtain CTC+EMT test results. After 10 weeks of treatment with leronlimab, this patients CTC+EMT results were zero (results reported on 2/12/2020).

Patient #3: Enrolled on 1/3/2020. This patients CAML counts went down from 45 to 30. CTC+EMT are stable and there has been no change in the total number.

Patient #4: Enrolled on 1/7/2020. This patients total CTC+EMT dropped by 75% in the first two weeks of treatment with leronlimab.

Patient #5: Enrolled on 2/4/2020. This patients CTC+EMT have been recorded upon enrollment and the first results are expected on 2/25/2020.

In addition to the first five patients, enrollment and treatment updates in CytoDyns Phase 2 protocol basket trial under its cancer IND are as follows:

Patient #6: Injected on 2/8/2020 and the first results since enrollment are due by end of February.

Patient #7: Injected on 2/13/2020.

Patients #8, 9 and 10: Completed screening for enrollment.

The patients enrolled in the mTNBC Phase 1b/2 trial continue to demonstrate meaningful results that support the hypothesis regarding leronlimabs mechanism of action, said Bruce Patterson, M.D., chief executive officer and founder of IncellDx, a diagnostic partner and an advisor to CytoDyn. In the four patients (1 with MBC, 3 with TNBC) now with results from leronlimab therapy, patients #1-3 have zero CTCs and zero EMTs and Patient #4, who has been treated with leronlimab for 2 weeks showed a decrease of CTCs and EMTs from 8 to 2. New data from Patient #2 with Stage 4 MBC and who has been treated with 10 weekly doses of leronlimab showed zero CTCs and zero EMTs, in addition to the shrinkage or disappearance of some brain metastases as previously reported.

Nader Pourhassan, Ph.D., president and chief executive officer of CytoDyn, added: These findings are extremely promising in light of the success rate of other treatment options. Therapeutic options for patients suffering from breast cancer are highly limited and we look forward to continuing enrollment and exploring leronlimabs potential to treat this devastating disease. Since our basket trial for all solid tumor cancers has been initiated, we are currently screening a prostate cancer patient, and if continued positive clinical results are forthcoming from this patient, we are hopeful that this will clear the path for CytoDyn to file for Breakthrough Therapy designation for all solid tumor cancers. Our mechanism of action is not only focused on the inhibition of metastasis of solid tumor cancers, but also targets the tumor itself through macrophages, angiogenesis and T-reg.

About Triple-Negative Breast CancerTriple-negative breast cancer (TNBC) is a type of breast cancer characterized by the absence of the three most common types of receptors in the cancer tumor known to fuel most breast cancer growthestrogen receptors (ER), progesterone receptors (PR) and the hormone epidermal growth factor receptor 2 (HER-2) gene. TNBC cancer occurs in about 10 to 20 percent of diagnosed breast cancers and can be more aggressive and more likely to spread and recur. Since the triple-negative tumor cells lack these receptors, common treatments for breast cancer such as hormone therapy and drugs that target estrogen, progesterone, and HER-2 are ineffective.

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) have granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases including NASH. Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is therefore conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additionalclinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in the first quarter of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients and plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSMedia:Grace FotiadesLifeSci Communicationsgfotiades@lifescicomms.com(646) 876-5026

Investors: Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

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CytoDyn Reports Continued Positive Clinical Data on its Phase 1b/2 mTNBC and Expanded Access Studies for MBC Ahead of Breakthrough Therapy Designation...

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Autologous Stem Cell And Non-Stem Cell Based Therapies Market 2020-2025 New Updates || Leadinf Players Fibrocell, Genesis Biopharma, Georgia Health...